Choice of recall period for patient-reported outcome (PRO) measures: criteria for consideration.

PURPOSE: Understand the choice of recall period for PRO measures based on intended use, characteristics of the disease, treatment, and attributes of studies. METHODS: Current practice and considerations were reviewed within several disease areas (overactive bladder, menopausal hot flashes, niacin-induced flushing, osteoarthritis pain, irritable bowel symptoms, benign prostatic hyperplasia, and alopecia). RESULTS: Rationales were identified for using different recall periods, including event-driven (immediate), daily, up to weekly, and longer than weekly. This work demonstrates that (1) recall depends on what the PRO measure captures, its intended use, and attributes of the disease and study; (2) within the same disease area, recall can vary depending on the concept or phenomenon of interest; (3) recall must consider patient burden and their ability to easily and accurately recall the information requested; and (4) recall must be consistent with the duration of the trial and the scheduled clinic visits. CONCLUSIONS: Shorter recall periods may underestimate symptom burden when symptoms have diurnal or day-to-day fluctuation and may place undue burden on patients. On the other hand, recall intervals that are too long may either over- or underestimate the health state. Therefore, appropriate criteria should be considered given attributes of the disease when selecting an adequate recall period.

Evaluating patient-reported outcome measurement comparability between paper and alternate versions, using the lung function questionnaire as an example.

OBJECTIVES: The goal of this study was to provide recommended steps to assess measurement comparability using a crossover study design and to demonstrate these steps using a short patient-reported outcome (PRO) instrument as an example. METHODS: The example PRO instrument was administered via paper, Web, interactive voice response system, and interview; a randomized crossover design was used to gather data across the multiple administration types. Participants completed the PRO instrument, demographic and health questions, and a short preference questionnaire. Evaluation included comparisons of the item-level responses and agreement, comparison of mean scale scores, score classifications, and questions designed to collect usability and administration preference. Here the authors provide a four-step evaluation guide to evaluate measurement comparability and illustrate these steps using a case-finding tool. RESULTS: In the example, item-level kappa statistics between the paper and the alternate versions ranged from good to excellent, intraclass correlation coefficient for mean scores were above 0.70, and the rate of disagreement ranged from 2% to 14%. In addition, although participants had an administration preference, they reported few difficulties with the versions they were assigned. CONCLUSIONS: The steps described in this article provide a guide for evaluating whether to combine scores across administration versions to simplify analyses and interpretation under a crossover design. The guide recommends the investigation of item-level responses, summary scores, and participant usability/preference when comparing versions, with each step providing unique information to support comprehensive evaluation and informed decisions regarding whether to combine data.

The impact of rotavirus gastroenteritis on the family.

BACKGROUND: Rotavirus is the leading cause of severe diarrhea in young children and causes substantial morbidity and mortality. Although the clinical aspects have been well described, little information is available regarding the emotional, social, and economic impact of rotavirus gastroenteritis on the family of a sick child. The objectives of this study were to: 1) assess the family impact of rotavirus gastroenteritis through qualitative interviews with parents; 2) compare the clinical severity of rotavirus-positive and negative gastroenteritis; 3) test a questionnaire asking parents to rank the importance of various factors associated with a case of rotavirus gastroenteritis. METHODS: The study enrolled parents and children (2-36 months of age) brought to one of the study sites (outpatient clinic or ER) if the child experienced > or = 3 watery or looser-than normal stools and/or forceful vomiting within any 24-hour period within the prior 3 days. The clinical severity of each child's illness was rated using a clinical scoring system and stool samples were tested for rotavirus antigen. Parents of rotavirus-positive children were invited to participate in focus group or individual interviews and subsequently completed a questionnaire regarding the impact of their child's illness. RESULTS: Of 62 enrolled children, 43 stool samples were collected and 63% tested positive for rotavirus. Illness was more severe in children with rotavirus-positive compared to rotavirus-negative gastroenteritis (92% vs. 37.5% rated as moderate/severe). Seventeen parents of rotavirus-positive children participated in the interviews and completed the written questionnaire. Parents were frightened by the severity of vomiting and diarrhea associated with rotavirus gastroenteritis, and noted that family life was impacted in several ways including loss of sleep, missed work, and an inability to complete normal household tasks. They expressed frustration at the lack of a specific medication and the difficulty of treating the illness with oral rehydration solutions, but had a largely positive outlook concerning the prospect of a rotavirus vaccine. CONCLUSION: A better understanding of how rotavirus gastroenteritis impacts the family can help healthcare providers ease parental fears and advise them on the characteristics of this illness, practices to prevent infection, and the optimal care of an affected child.

Validation of the Knee Injury and Osteoarthritis Outcome Score subscales for patients with articular cartilage lesions of the knee.

BACKGROUND: The Knee Injury and Osteoarthritis Outcome Score (KOOS) assesses acute and chronic knee injuries or early-onset osteoarthritis in young, active patients. The United States Food and Drug Administration guidelines recommend that patient-reported outcome instruments used to support clinical trial label claims should demonstrate content validity using patient input and have acceptable psychometric properties in the target population. To use the KOOS subscales in safety and efficacy trials assessing new treatments for patients with articular cartilage lesions, additional validation work, using input from patients with articular cartilage lesions, was necessary. PURPOSE: Qualitative and quantitative evaluations of the KOOS subscales' validity among patients with articular cartilage lesions were conducted to support their use as clinically meaningful end points in clinical trials. STUDY DESIGN: Cohort study (diagnosis); Level of evidence, 2. METHODS: For qualitative analysis, cognitive interviews involving concept elicitation and cognitive debriefing with the KOOS items were conducted with 15 participants aged 25 to 52 years. Participants either were candidates for cartilage repair or had undergone cartilage repair 6 months or more before the study. For the quantitative analysis, a psychometric evaluation of the KOOS was conducted with clinical trial data from 54 patients, aged 18 to 55 years, evaluating the Cartilage Autograft Implantation System in the United States (n = 29) and the European Union (n = 25). Data were collected before surgery and at 7 postsurgical visits up to 12 months. Internal consistency and test-retest reliability, construct validity, responsiveness, and estimates of the minimal detectable change (MDC) were assessed. Test-retest reliability was assessed using data from months 2 and 3 on a subset of stable patients. RESULTS: Qualitative research confirmed that concepts measured on the KOOS are important to patients with articular cartilage lesions. Most participants reported the KOOS was comprehensive and appropriate. In the quantitative research, KOOS subscales showed excellent internal consistency reliability (range, .74-.97 at baseline) and test-retest reliability (range, .78-.82). Construct validity results supported hypothesized relationships, with significant correlations (r ≥ .50) in the expected directions. Responsiveness analyses demonstrated excellent sensitivity to change; standardized response means ranged from 0.8 to 1.2, and MDC estimates ranged from 7.4 to 12.1. CONCLUSION: The study results support the use of the KOOS subscales among patients with articular cartilage lesions.

Validation of alternate modes of administration of the lung function questionnaire (LFQ) in subjects with smoking history.

PURPOSE: The lung Function Questionnaire (LFQ) was developed and validated as a case-finding tool to identify patients at risk of airflow obstruction (AO) that should be evaluated further using spirometry. Our objective was to assess the usability and validity of additional questionnaire-administration modes, including Web-based, interactive voice response system (IVRS)-based, and interviewer-based modes. DESIGN: This multicenter, prospective, noninterventional data-collection study enrolled 149 individuals aged ≥40 years with current or former smoking history. A two-visit crossover design was employed; patients completed the paper-based LFQ and were randomly assigned to complete one of three alternate modes. METHODS: Statistical evaluation included item-level, scale-level, and AO risk-classification comparisons; a satisfaction survey assessed patient preference. RESULTS: this study showed a great degree of concordance between alternate forms of the LFQ and the paper version. Results indicated an absence of floor and ceiling effects and the average LFQ item-level means were consistent across modes. LFQ scores were stable between assessments, (administered approximately one week apart) showed exceptionally good agreement, and AO risk classification using the LFQ cut point was consistent across modes. CONCLUSIONS: The LFQ is an important case-finding tool to aid primary care physicians in further evaluating symptomatic patients at risk of AO. The alternate modes will further facilitate the implementation and widespread uptake of this tool.

Development and psychometric properties of the HPV Impact Profile (HIP) to assess the psychosocial burden of HPV.

OBJECTIVE: A comprehensive questionnaire designed to assess the full spectrum of potential human papillomavirus (HPV)-related psychosocial effects in women does not exist. The HPV Impact Profile (HIP) was developed to determine the psychosocial impact of HPV infection and related interventions. RESEARCH DESIGN AND METHODS: Draft instrument items and domains were developed using a literature review and cognitive debriefing interviews with women who had experienced HPV-related conditions. An importance rating questionnaire guided item ranking and reduction. A draft questionnaire was pilot-tested for comprehension and ease of completion. Psychometric evaluation of the final HIP was conducted in a survey of 583 women. Data quality, item acceptability, scale acceptability, reliability, and discriminate construct validity were assessed. OUTCOME MEASURE: The final HIP contained 29 items rated on a 0-10 point discretized visual analog scales grouped into seven hypothesized domains. RESULTS: Total HIP scores ranged from 0 (no impact) to 100 (worst impact). Data quality was high, with missing data for items ranging from 0 to 0.7% and over 99% of the scores were computable. Cronbach's alpha ranged from 0.64 to 0.90 and was > or =0.7 for 5/7 domains. Discriminant construct validity was demonstrated. Appropriate modifications could potentially be made to improve some aspects of the HIP, including modification to include other HPV diseases such as head and neck, anal, and vulvovaginal cancers and HPV disease in men. CONCLUSIONS: The disease-specific HIP has favorable reliability and construct validity and a good ability to discriminate among disease severity.

Validation of the nighttime symptoms score as a clinically relevant measure of allergic rhinitis.

The aim of this study was to validate the nighttime symptoms score (NSS), which incorporates individual scores for difficulty going to sleep and nighttime awakening caused by nasal symptoms and nasal congestion on awakening, as a clinically relevant measure of allergic rhinitis (AR). Fifty-five general season AR (SAR) symptom items were generated by interviews with 14 patients with symptomatic SAR without concomitant asthma for use in an Importance Rating Questionnaire (IRQ). A second group of patients (n = 83) with symptomatic AR without asthma rated the importance of each item on the IRQ. Correlation coefficients were calculated to examine the relationships between the six sleep quality questions on the IRQ and the other AR symptoms and between the symptom questions of the NSS, the Daytime Nasal Symptoms Score (DNSS), and the individual domains of the Rhinoconjunctivitis Quality-of-Life Questionnaire (RQLQ). The majority (94%) of patients with active AR reported some degree of symptoms relating to sleep quality. The six sleep quality items on the IRQ were selected by 71-84% of patients. The sleep quality items were more highly correlated with each other (r = 0.48-0.85) than with the four items of the DNSS (r = 0.01-0.42). There was a moderate-to-strong correlation of the RQLQ sleep domain with the two sleep questions of the NSS (r = 0.44-0.57). The individual symptom questions of the NSS and the DNSS were only moderately correlated with each other. Sleep quality questions measure aspects of SAR that are not captured by daytime SAR symptoms. The results show that the NSS is a valid and relevant clinical measure of the impact of nighttime sleep disturbance on AR patients.